Diseases related to immunoglobulins and immunoglobulin-producing plasma cells include clonal plasma cell dyscrasias and autoimmune diseases.
Plasma cell dyscrasias including but not limited to monoclonal gammopathy of undetermined significance (MGUS), symptomatic and smoldering multiple myeloma, Waldenstrom's macroglobulinemia, and systemic light-chain amyloidosis are derived from a single immortalized founding clone and produce a clonal immunoglobulin protein. Both the immunoglobulin proteins and the clonal plasma cells in these diseases can damage organs such as the heart, kidneys, liver, gastrointestinal tract, bones, immune system, thyroid, soft tissues and peripheral nervous system.
Existing therapies include steroids, toxic chemotherapy agents such as melphalan, adriamycin and cyclophosphamide, as well proteasome inhibitors, immunomodulatory drugs, radiation, and autologous and allogeneic stem cell transplantation. These diseases remain incurable and shorten survival.
Autoimmune diseases arise from an inappropriate immune response of the body against substances and tissues normally present in the body. Autoimmune diseases are characterized by the destruction of one or more types of body tissue, abnormal growth of an organ, or changes in organ function. An autoimmune disorder may affect one or more organ or tissue types. Organs and tissues commonly affected by autoimmune disorders include: blood vessels, connective tissues, endocrine glands such as the thyroid or pancreas, joints, muscles, red blood cells, or skin.
A person may have more than one autoimmune disorder at the same time. Examples of autoimmune (or autoimmune-related) disorders include, but are not limited to, systemic lupus erythematosus, type I diabetes, Addison's disease, celiac disease, dermatomyositis, Graves disease, Hashimoto's thyroiditis, multiple sclerosis, myasthenia gravis, pernicious anemia, reactive arthritis, rheumatoid arthritis, and Sjogren syndrome.
Existing treatments include supplements to replace a hormone or vitamin that the body is lacking (e.g., thyroid supplements, vitamins such as B12, or insulin injections), blood transfusions, and immunosuppressive medicines. Such medicines may include corticosteroids (such as prednisone) and nonsteroid drugs such as azathioprine, cyclophosphamide, mycophenolate, sirolimus, or tacrolimus.
Existing treatments can have serious side effects and are not always or often effective. New therapies are needed.